Compositions and methods for the treatment and prevention of vascular malformations
AssigneeThe Trustees of Columbia University in the City of New York
InventorsCarrie J. Shawber, June K. Wu, Ajit Muley
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Abstract
Provided herein are methods and compositions for treating and preventing vascular malformations including lymphatic, venous, capillary, arteriovenous, and combinations thereof. Methods of treatment and prevention include the administration of proteasome inhibitors, omipalisib, disulfiram, and agents which target genes in the PI3K/AKT/mTOR or RAS/RAF/MAPK pathways, including but not limited to Pik3ca, Pik3r3, Tsc2, Rasa1, Map2k2, and Glmn.
Claims42
A method of treating or preventing a vascular malformation in a subject in need thereof comprising administering to the subject a therapeutically effective amount of a proteasome inhibitor or an agent or compound with proteasome inhibiting ability.
A method of treating or preventing a vascular malformation in a subject in need thereof comprising administering to the subject a therapeutically effective amount of disulfiram.
A method of treating or preventing a vascular malformation in a subject in need thereof comprising administering to the subject a therapeutically effective amount of omipalisib.
A method of treating or preventing a vascular malformation in a subject in need thereof comprising administering to the subject a therapeutically effective amount of an agent which targets a mutation in a gene chosen from the group consisting of Pik3ca, Pik3r3, Tsc2, Rasa1, Map2k2, Glmn, and combinations thereof.
A method of treating or preventing a vascular malformation in a subject in need thereof comprising administering to the subject a therapeutically effective amount of an agent which targets a mutation in a gene chosen from the group consisting of genes in the PI3K/AKT/mTOR or RAS/RAF/MAPK pathway.
A method of treating or preventing a vascular malformation in a subject in need thereof comprising administering to the subject a therapeutically effective amount of a copper-binding agent or compound.
Dependencies are inferred from the claim text.